Stealth BioTherapeutics Corp. (NASDAQ:MITO) was founded in 2006 and is headquartered in Newton, Massachusetts, USA, with 57 full-time employees (12/31/2018). It is a clinical-stage biotechnology company focusing on diseases involving mitochondrial dysfunction. For the discovery, development and commercialization of new therapies, its wholly-owned subsidiaries include: Delaware company Stealth BioTherapeutics, Inc., Hong Kong Stealth BioTherapeutics (HK) Limited, China Stealth BioTherapeutics (Shanghai) Limited.
Stealth BioTherapeutics (MITO):
Mitochondria exist in almost every cell of the body. They are the main source of body energy production and are essential for normal organ function. Dysfunctional mitochondria is a feature of many rare genetic diseases, collectively referred to as primary mitochondrial diseases, and many common age-related diseases are also involved. Stealth BioTherapeutics believes that its main product candidate elamipretide may treat rare genetic and common age-related mitochondrial diseases. Stealth BioTherapeutics’ mission is to become a leader in mitochondrial medicine. The company has established an experienced management team, board of directors and scientific advisory team to help it achieve this mission.
Stealth BioTherapeutics is mainly focused on discovering and developing new first-in-class therapies for diseases related to mitochondrial dysfunction. Elamipretide is its main product candidate.
The wonderful compound of Elamipretide developed by Stealth BioTherapeutics is used in primary mitochondrial myopathy, heart failure, dry age-related macular degeneration, Pap syndrome, Huntington’s disease, acute kidney injury, heterochromatic iridocyclitis, and Leber inheritance. A series of indications such as sexual optic neuropathy have also shown good results. The related results have been published in nearly 100 academic journals. At the same time, many indications have been approved by the US FDA to enter clinical trials.
Stealth BioTherapeutics is studying elamipretide in the following indications:
- Primary mitochondrial myopathy -characterized by weak skeletal muscle weakness, exercise intolerance and fatigue, the company believes this affects an estimated 40,000 patients in the United States. None of the US Food and Drug Administration (FDA), European Medicines Agency (EMA), or China National Medical Products Association (NMPA) has approved therapies to treat this indication. Stealth BioTherapeutics has received Fast Track (fast approval) and Orphan Drug (orphan drug) designations from the FDA for the development of elamipretide in this indication. The company is conducting Phase III key trials in North America and Europe and expects to obtain data from this trial by the end of 2019.
- Barth syndrome -is characterized by myocardial weakness, cardiomyopathy, neutropenia or low white blood cell count (which may increase the risk of infection), skeletal muscle weakness, growth retardation, fatigue and various degrees of physical disability. It is estimated that in the United States, the incidence of the disease is between 1 in 300,000 to 1 in 400,000, and there are approximately 200 surviving patients worldwide. The FDA, EMA or NMPA have not approved therapies for the treatment of Barth. Stealth BioTherapeutics has received Fast Track and Orphan Drug designations from the FDA for the development of elamipretide in this indication. In December 2018, the company completed the placebo-controlled trial of the Phase II/III clinical trial of Barth patients. The company plans to meet with the FDA in the first half of 2019 to discuss potential new drug application (NDA) submissions.
- Leber hereditary optic neuropathy (LHON)-characterized by loss of central vision. It is estimated that LHON affects approximately 10,000 people in the United States, and an estimated 70% of them have the genetic mutation G11778A that the company is studying. The FDA or NMPA has not approved therapies for the treatment of LHON, and EMA has only one approved therapy. Stealth BioTherapeutics has received Fast Track and Orphan Drug designations from the FDA for the development of elamipretide in this indication. The company completed the placebo-controlled trial of phase II clinical trials in the first half of 2018. Although the trial did not reach its primary endpoint of best-corrected visual acuity changes, a trend that favored elamipretide across multiple endpoints was observed. It is planned to meet with the FDA in the first half of 2019 for an end-of-Phase 2 meeting. After the meeting, the company plans to submit a clinical trial application to NMPA in order to include clinical trial sites in China into potential phase III clinical trials.
- Dry Acute Macular Degeneration (Dry AMD) -It is characterized by visual distortion, low vision, decreased vision, overall vision loss, and blurred vision. It is estimated to affect more than 10 million people in the United States and is the main cause of blurred vision. No FDA, EMA or NMPA has approved therapies for the treatment of dry AMD. Stealth BioTherapeutics completed a phase I trial of patients with drusen (an early form of dry AMD) and geographic atrophy (an advanced form of dry AMD), in which the company observed various visual function parameters of drusen and geographic location Statistically significant improvement from baseline. The company was granted Fast Track designation from the FDA in November 2018 for the development of elamipretide for dry AMD patients with geographic atrophy. The company plans to launch a Phase IIb clinical trial in the first quarter of 2019 to treat patients with geographic atrophy.
In addition to Stealth BioTherapeutics’ clinical development plan for elamipretide, the company also plans to evaluate SBT-20, the company’s second clinical stage candidate product for the treatment of rare diseases, such as peripheral neuropathies (peripheral neuropathies).
Stealth BioTherapeutics is also developing SBT-272, a pre-clinical product candidate for rare neurodegenerative diseases.
Stealth BioTherapeutics (MITO) investment:
Stealth BioTherapeutics Corp. (NASDAQ:MITO) submitted its IPO prospectus on 12/28/2018, and listed on Nasdaq on 2/15/2019 IPO, with an issue price of US$12.00, issuing 6.2 million shares, raising US$74.4 million, Jefferies / Evercore ISI/ BMO Capital Markets underwriting.